Understanding RNA dysfunction using novel RNA-seq technologies

To gain a better understanding of the underlying mechanisms of the faulty TDP-43 and FUS proteins and how they might drive the disease.

Sam Bryce Smith Cropped
Pietro Fratta

PhD Student: Samuel Bryce-Smith

Principal Investigator: Professor Pietro Fratta

Lead Institution: University College London

MND Association Funding: £100,000*

Funding dates: November 2019 - December 2022

*Supported by The Masonic Charitable Foundation PhD Studentship

About the project

Correct processing of RNA (molecules that carry information to make proteins from DNA) is crucial for survival. Proteins called TDP-43 and FUS are important for the processing of RNA. This project will help understand how changes in TDP-43 and FUS impact on motor neuron RNA and survival. Molecular changes occurring in early stages of disease can be identified using RNA-sequencing (RNA-seq) technology.  This knowledge will be invaluable for developing effective therapeutics and the project will allow application of cutting-edge technology to numerous MND mouse and human cell models.

What does this mean for people living with MND?

This project will contribute to understanding how changes in TDP-43 and FUS impact on motor neuron RNA and survival. The results of this could provide invaluable knowledge for the development of new therapies for MND and provide further insights into changes occurring during the early stages of disease.


Want to find out more about this project? Check out the resources below:


Panic at the (Cell's) Depot 

Project code: 893-792